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Blood Cells Mol Dis. 2014 Jun-Aug;53(1-2):56-60. doi: 10.1016/j.bcmd.2014.02.006. Epub 2014 Feb 25.

Impact of velaglucerase alfa on bone marrow burden score in adult patients with type 1 Gaucher disease: 7-year follow-up.

Author information

1
Gaucher Clinic, Shaare Zedek Medical Center, The Hebrew University-Hadassah School of Medicine, Jerusalem, Israel. Electronic address: elstein@szmc.org.il.
2
Department of Radiology, Yale University School of Medicine, New Haven, CT, USA.
3
Shire, Lexington, MA, USA.
4
Gaucher Clinic, Shaare Zedek Medical Center, The Hebrew University-Hadassah School of Medicine, Jerusalem, Israel.

Abstract

BACKGROUND:

Bone marrow infiltration by substrate-engorged "Gaucher" cells manifests early in Gaucher disease (GD). The impact of velaglucerase alfa on bone marrow burden (BMB) was evaluated as an exploratory assessment.

METHODS:

BMB scores were assessed using T1- and T2-weighted magnetic resonance images of the lumbar spine (LS) and femora among symptomatic GD patients who participated in the 9-month Phase I/II trial and long-term extension study for velaglucerase alfa. A post-hoc assessment of marrow involvement was performed. BMB scores per site are 0-8 (0/1=normal; 8=severe infiltration).

RESULTS:

The median LS-BMB score at baseline was 6 (n=12; range 3-8); at 9 months, compared with baseline, there was a median change of -2 (n=11; two-sided p-value=0.0078). LS-BMB scores continued to decrease through 5 years (n=8; median change from baseline -5 [p=0.0078], median score 1 [range 1-4]) and were subsequently sustained through 7 years (n=8). LS-BMB decreases of ≥2 points occurred in 6/11 patients at 9 months, and in all assessable patients (8/8) by 5 years. Long-term femoral BMB (F-BMB) assessment was possible for three patients; all experienced reductions of ≥2 points at 5 years with a total score (LS-BMB+F-BMB) decrease ≥4.

CONCLUSIONS:

This post hoc analysis suggests improvement in BMB scores through 5 years that was sustained through 7 years, despite dose reduction from 15 months. Prospective studies in a large cohort are needed to validate these findings.

KEYWORDS:

Bone marrow burden; Bone mineral density; Enzyme replacement therapy; Gaucher disease; MRI; Velaglucerase alfa

PMID:
24581483
DOI:
10.1016/j.bcmd.2014.02.006
[Indexed for MEDLINE]
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